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  1. Book: New findings on inhibitor development: from registries to clinical studies

    Peyvandi, Flora / Makris, Michael

    (Haemophilia ; volume 23, supplement 1 (January 2017))

    2017  

    Author's details guest editors: F. Peyvandi, M. Makris
    Series title Haemophilia ; volume 23, supplement 1 (January 2017)
    Collection
    Language English
    Size 13 Seiten, Diagramme
    Publisher Wiley Blackwell
    Publishing place Oxford, UK
    Publishing country Great Britain
    Document type Book
    HBZ-ID HT019283851
    Database Catalogue ZB MED Medicine, Health

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    Zs A 4249 -23,Suppl.1- not available for loan Zeitschriften-Lesesaal

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  2. Article ; Online: Laboratory Testing for von Willebrand Factor Activity by a Glycoprotein Ib-Binding Assay (VWF:GPIbR): HemosIL von Willebrand Factor Ristocetin Cofactor Activity on ACL TOP

    Seidizadeh, Omid / Peyvandi, Flora

    Methods in molecular biology (Clifton, N.J.)

    2023  Volume 2663, Page(s) 669–677

    Abstract: von Willebrand disease (VWD) is a lifelong and common inherited bleeding disorder caused by a quantitative deficiency and/or qualitative defect of von Willebrand factor (VWF). In order to establish the correct diagnosis of VWD, various tests must be ... ...

    Abstract von Willebrand disease (VWD) is a lifelong and common inherited bleeding disorder caused by a quantitative deficiency and/or qualitative defect of von Willebrand factor (VWF). In order to establish the correct diagnosis of VWD, various tests must be conducted, including evaluation of factor VIII activity (FVIII:C), VWF antigen (VWF:Ag), and VWF functional activity. The platelet-dependent VWF activity is measured in different ways, with the historical ristocetin cofactor assay (VWF:RCo) using platelet aggregometry now replaced with newer assays that offer better precision, lower limits of detection, low coefficient of variation, and are fully automated. The VWF activity by glycoprotein Ib-binding assays (VWF:GPIbR) measured on the ACL TOP
    MeSH term(s) Humans ; von Willebrand Factor ; Platelet Glycoprotein GPIb-IX Complex ; von Willebrand Diseases/diagnosis ; Blood Coagulation Tests ; Sensitivity and Specificity
    Chemical Substances von Willebrand Factor ; Platelet Glycoprotein GPIb-IX Complex
    Language English
    Publishing date 2023-05-18
    Publishing country United States
    Document type Journal Article
    ISSN 1940-6029
    ISSN (online) 1940-6029
    DOI 10.1007/978-1-0716-3175-1_44
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  3. Article ; Online: Ultrasound evaluation of hemophilic arthropathy: a proposal of definitions in a changing landscape.

    Gualtierotti, Roberta / Solimeno, Luigi Piero / Peyvandi, Flora

    Research and practice in thrombosis and haemostasis

    2024  Volume 8, Issue 1, Page(s) 102314

    Abstract: The advent of novel effective treatments and the identification of the need to achieve a higher trough level for persons with hemophilia A and B have changed the landscape of management of these patients, allowing to change the target from survival and ... ...

    Abstract The advent of novel effective treatments and the identification of the need to achieve a higher trough level for persons with hemophilia A and B have changed the landscape of management of these patients, allowing to change the target from survival and prevention of life-threatening complications to prevention of musculoskeletal complications and improvement of quality of life. Point-of-care musculoskeletal ultrasound imaging has also improved the early recognition of joint bleeding and the differential diagnosis of acute joint pain. In addition, joint ultrasound allows the evaluation of the severity of hemophilic arthropathy in terms of synovitis and cartilage and bone damage. However, a lack of standardization in the definition of ultrasound elementary lesions of hemophilic arthropathy may lead to confusion and an incorrect evaluation of the presence and progression of joint damage. Here, we propose to start a standardization and validation process for ultrasound definitions of hemophilic arthropathy that has been planned to become a project within the Factor VIII/IX Standardization Subcommittee of the International Society on Thrombosis and Haemostasis Scientific and Standardization Committee.
    Language English
    Publishing date 2024-01-03
    Publishing country United States
    Document type Editorial
    ISSN 2475-0379
    ISSN (online) 2475-0379
    DOI 10.1016/j.rpth.2023.102314
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  4. Article ; Online: Phase 3 study of recombinant von Willebrand factor in patients with severe von Willebrand disease who are undergoing elective surgery: Reply.

    Peyvandi, Flora

    Journal of thrombosis and haemostasis : JTH

    2019  Volume 17, Issue 8, Page(s) 1405–1406

    MeSH term(s) Elective Surgical Procedures ; Factor VIII ; Humans ; von Willebrand Diseases ; von Willebrand Factor
    Chemical Substances von Willebrand Factor ; Factor VIII (9001-27-8)
    Language English
    Publishing date 2019-09-08
    Publishing country England
    Document type Letter ; Comment
    ZDB-ID 2112661-6
    ISSN 1538-7836 ; 1538-7933
    ISSN (online) 1538-7836
    ISSN 1538-7933
    DOI 10.1111/jth.14545
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    Zs.A 5805: Show issues Location:
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    Zs.MO 295: Show issues

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  5. Article ; Online: ISTH's new strategic plan 2024-2028: A transformative journey toward shaping the future of thrombosis and hemostasis.

    Peyvandi, Flora / Angchaisuksiri, Pantep / Reiser, Thomas

    Journal of thrombosis and haemostasis : JTH

    2024  

    Language English
    Publishing date 2024-04-25
    Publishing country England
    Document type Editorial
    ZDB-ID 2112661-6
    ISSN 1538-7836 ; 1538-7933
    ISSN (online) 1538-7836
    ISSN 1538-7933
    DOI 10.1016/j.jtha.2024.04.013
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    Zs.MO 295: Show issues

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  6. Article ; Online: Air pollution and cardiovascular health in Middle East and North Africa: many shadows but some light.

    Mannucci, Pier Mannuccio / Peyvandi, Flora

    European journal of preventive cardiology

    2022  Volume 30, Issue 3, Page(s) 254–255

    MeSH term(s) Humans ; Cardiovascular Diseases/diagnosis ; Cardiovascular Diseases/epidemiology ; Particulate Matter/adverse effects ; Global Burden of Disease ; Middle East/epidemiology ; Africa, Northern/epidemiology ; Air Pollution/adverse effects
    Chemical Substances Particulate Matter
    Language English
    Publishing date 2022-11-10
    Publishing country England
    Document type Editorial ; Comment
    ZDB-ID 2626011-6
    ISSN 2047-4881 ; 2047-4873
    ISSN (online) 2047-4881
    ISSN 2047-4873
    DOI 10.1093/eurjpc/zwac292
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    Zs.A 4686: Show issues Location:
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  7. Article ; Online: ISTH Biennial Impact Report: Looking back and looking forward.

    Weitz, Jeffrey I / Peyvandi, Flora

    Journal of thrombosis and haemostasis : JTH

    2022  Volume 20, Issue 7, Page(s) 1515–1517

    Language English
    Publishing date 2022-07-11
    Publishing country England
    Document type Editorial
    ZDB-ID 2112661-6
    ISSN 1538-7836 ; 1538-7933
    ISSN (online) 1538-7836
    ISSN 1538-7933
    DOI 10.1111/jth.15749
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  8. Article ; Online: Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies.

    Di Minno, Giovanni / Miesbach, Wolfgang / Castaman, Giancarlo / Peyvandi, Flora

    Haematologica

    2024  

    Abstract: Three major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for ...

    Abstract Three major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models reflecting the heterogeneity of certain clinical settings (e.g., congestive heart failure) are poorly available for improving AAV-based GT. Finally, antisense and gene editing approaches will soon complement gene augmentation strategies for the stable solution of unsolved issues of AAV-based GT. While minimizing toxicity, next-generation AAV vectors should decrease the viral load needed to achieve therapeutic efficacy; be functional in a restricted cellular subset; avoid transgene expression in unwanted cells (e.g., hepatocytes), and escape immune oversight in AAV-based GT. The role of stress-induced apoptosis in the loss of transgene expression in GT should be also explored. Aligning interests and obligations of pharmaceutical industry with those of the health system is critical for AAV-based GT success. Costs and challenges for pharmaceutical industry include a) removing impurities from AAV; b) validating tests to measure treatment efficacy, c) promoting training programs to standardize vector genomes delivery, d) collecting long-term follow-up data, and e) maintaining sustainability and cost-effectiveness of AAV-based GT. In rare disorders with small patient numbers (e.g., hemophilia), clearcut outcomes are mandatory as endpoints of unequivocal efficacy data.
    Language English
    Publishing date 2024-03-07
    Publishing country Italy
    Document type Journal Article
    ZDB-ID 2333-4
    ISSN 1592-8721 ; 0017-6567 ; 0390-6078
    ISSN (online) 1592-8721
    ISSN 0017-6567 ; 0390-6078
    DOI 10.3324/haematol.2023.284622
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  9. Article ; Online: [No title information]

    Casini, Alessandro / Al-Samkari, Hanny / Hayward, Catherine / Peyvandi, Flora

    Haemophilia : the official journal of the World Federation of Hemophilia

    2024  Volume 30 Suppl 3, Page(s) 60–69

    Abstract: Inherited factor coagulation deficiencies and vascular bleeding disorders, associated with bleeding of various severity, are often classified as rare bleeding disorders (RBDs). These include inherited fibrinogen disorders, inherited platelet function ... ...

    Title translation Rare bleeding disorders: Advances in management.
    Abstract Inherited factor coagulation deficiencies and vascular bleeding disorders, associated with bleeding of various severity, are often classified as rare bleeding disorders (RBDs). These include inherited fibrinogen disorders, inherited platelet function disorders (IPFD) and hereditary haemorrhagic telangiectasia (HHT). In the last decades, there have been large increases in knowledge on the epidemiology, genetics, physiopathology, clinical features, and diagnosis of RBDs, but improvements in management have been more limited and remain challenging. The treatment mainstay of RBDs is based only on replacement of a few available coagulation factor concentrates or cryoprecipitates. There is growing interest in therapeutic agents that enhance coagulation or inhibiting anticoagulant pathways in RBDs. In severe IPFD, the optimal platelet transfusion strategy is not yet established. Moreover, data is scarce on the effectiveness and safety of desmopressin and/or antifibrinolytic drugs often used for milder IPFD treatment. The best fibrinogen replacement strategy (prophylaxis vs. on demand) in afibrinogenemia is still debated. Similarly, the optimal trough fibrinogen target level for treatment of acute bleeding, and the role of fibrinogen replacement during pregnancy in mild hypofibrinogenemia and dysfibrinogenemia, have not been properly evaluated. The therapeutic arsenal in HHT includes antifibrinolytics and a series of antiangiogenic agents whose potential efficacy has been tested in small studies or are under investigation for treatment of bleeding. However, there is need to address several issues, including the optimal dosing strategies, the potential emergent toxicity of longer-term use, and the impact of systemic antiangiogenic treatment on visceral arteriovenous malformations.
    MeSH term(s) Pregnancy ; Female ; Humans ; Blood Coagulation Disorders/drug therapy ; Hemorrhage/etiology ; Hemorrhage/prevention & control ; Fibrinogen/therapeutic use ; Blood Coagulation Factors/therapeutic use ; Afibrinogenemia/diagnosis ; Antifibrinolytic Agents/therapeutic use
    Chemical Substances Fibrinogen (9001-32-5) ; Blood Coagulation Factors ; Antifibrinolytic Agents
    Language Dutch
    Publishing date 2024-03-18
    Publishing country England
    Document type English Abstract ; Journal Article
    ZDB-ID 1229713-6
    ISSN 1365-2516 ; 1351-8216 ; 1355-0691
    ISSN (online) 1365-2516
    ISSN 1351-8216 ; 1355-0691
    DOI 10.1111/hae.14986
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    Zs.MO 450: Show issues

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  10. Article: Laboratory Diagnosis of Antiphospholipid Syndrome in Anticoagulated Patients.

    Tripodi, Armando / Scalambrino, Erica / Clerici, Marigrazia / Peyvandi, Flora

    Biomedicines

    2023  Volume 11, Issue 6

    Abstract: The laboratory diagnosis of antiphospholipid syndrome (APS) requires the measurement of solid-phase antibodies to cardiolipin or β2-Glycoprotein-I and the search for lupus anticoagulant (LA). The diagnosis of patients whilst on anticoagulation is ... ...

    Abstract The laboratory diagnosis of antiphospholipid syndrome (APS) requires the measurement of solid-phase antibodies to cardiolipin or β2-Glycoprotein-I and the search for lupus anticoagulant (LA). The diagnosis of patients whilst on anticoagulation is impaired by the difficult interpretation of results, at least for LA, owing to the fact that prolongations of clotting times induced by LA superimpose those induced by anticoagulants. This is a matter of concern as treating physicians very often need to know the APS status of their patients to make a decision on secondary antithrombotic prophylaxis. This article aims to review the effect brought about by anticoagulants on APS diagnosis and discuss the options that can be used to overcome such an effect.
    Language English
    Publishing date 2023-06-19
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2720867-9
    ISSN 2227-9059
    ISSN 2227-9059
    DOI 10.3390/biomedicines11061760
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